Category:

Dateline:

Public Company Information:

NASDAQ:

BIIB

US09062X1037

NASDAQ:

IONS

"As part of our mission to improve the lives of those affected by SMA, we remain steadfast in our commitment to work with healthcare professionals, advocacy groups and government agencies to ensure people who could benefit from SPINRAZA receive access to this important treatment as quickly as possible."

CAMBRIDGE, Mass.--(BUSINESS WIRE)--The European Commission (EC) has granted a marketing authorization for
SPINRAZA® (nusinersen) for the treatment of 5q spinal
muscular atrophy (SMA), Biogen
(NASDAQ:BIIB) announced today.1 5q SMA is the most common
form of the disease and represents approximately 95% of all SMA cases.2
SPINRAZA is the first approved treatment in the European Union (EU) for
SMA, a leading genetic cause of death in infants that is marked by
progressive, debilitating muscle weakness. SPINRAZA was reviewed under
the European Medicines Agency’s (EMA) accelerated assessment program,
intended to expedite access to patients with unmet medical needs.

“Today we join individuals and families affected by SMA across Europe in
celebrating the approval of SPINRAZA. Based on the robust efficacy and
safety profile demonstrated in the clinical trials, we believe SPINRAZA
will have a meaningful impact on infants, children and adults living
with this devastating disease,” said Michel Vounatsos, chief executive
officer at Biogen. “As part of our mission to improve the lives of those
affected by SMA, we remain steadfast in our commitment to work with
healthcare professionals, advocacy groups and government agencies to
ensure people who could benefit from SPINRAZA receive access to this
important treatment as quickly as possible.”

The approval of SPINRAZA is primarily based on results from two pivotal
multicenter, controlled studies, including end of study data from ENDEAR
(infantile-onset SMA) and an interim analysis of CHERISH (later-onset
SMA), both of which demonstrated the clinically meaningful efficacy and
favorable benefit-risk profile of SPINRAZA. The approval was also
supported by open-label data in pre-symptomatic and symptomatic
individuals with, or likely to develop, Types 1, 2 and 3 SMA.

In the ENDEAR end of study analysis, a statistically significant greater
percentage of patients achieved the definition of motor milestone
responder in the SPINRAZA group (51%) compared to the sham-control group
(0%) (p<0.0001). Some infants in the SPINRAZA group achieved motor
milestones including full head control, ability to roll, sitting, and
standing. Additionally, infants treated with SPINRAZA demonstrated a
statistically significant reduction (47%) in the risk of death or
permanent ventilation (p=0.0046).

In the CHERISH pre-specified interim analysis, there was a statistically
significant and clinically meaningful improvement in motor function in
children with later-onset SMA (most likely to develop Type 2 or Type 3)
treated with SPINRAZA compared to untreated children. Improvements were
measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) and
demonstrated a treatment difference of 5.9 points in the mean change
from baseline to Month 15 in the HFMSE score (p=0.0000002). The HFMSE is
a reliable and validated tool specifically designed to assess motor
function in children with SMA. The Phase 3 end of study data were
consistent with the interim analysis and presented at the American
Academy of Neurology annual meeting in Boston, Mass., April 2017.

“The overall clinical findings support the efficacy and safety of
SPINRAZA in a broad range of individuals with SMA, including significant
improvements in motor development and reduction in risk of death in
infants,” said Prof. Dr. Jan Kirschner from the Medical Center
University of Freiburg, Germany. “These unprecedented improvements bring
new hope to a community where there previously were no approved
treatments available to address the loss of motor function over time. We
are now seeing motor improvements with SPINRAZA that are never seen in
the natural course of the disease.”

SPINRAZA must be administered via intrathecal injection, which delivers
therapies directly to the cerebrospinal fluid (CSF) around the spinal
cord,3 where motor neurons degenerate in individuals with SMA
due to insufficient levels of survival motor neuron (SMN) protein.4

SPINRAZA demonstrated a favorable benefit-risk profile.
Thrombocytopenia, renal toxicity and coagulation abnormalities,
including acute severe thrombocytopenia, have been observed after
administration of other subcutaneously or intravenously administered
antisense oligonucleotides. There is a risk of adverse reactions
occurring as part of the lumbar puncture procedure (e.g. headache,
backpain, vomiting).

The timing of SPINRAZA availability in the EU will vary by country, per
local reimbursement and access pathways. Biogen has been working with
health systems and government agencies across the EU to help patients
secure access to SPINRAZA.

In 2016, in response to the urgent need for treatment for the most
severely affected individuals living with SMA, Biogen sponsored one of
the largest, pre-approval Expanded Access Programs (EAP) in rare disease
free of charge. The EAP has led to the initiation and ongoing treatment
of more than 350 eligible individuals with infantile-onset SMA (most
likely to develop Type 1) in 17 European countries.

SPINRAZA was first approved by the U.S. Food and Drug Administration
(FDA) on December 23, 2016 within three months of regulatory filing.
Biogen has also submitted regulatory filings in Japan, Canada,
Australia, Switzerland, and Brazil and plans to initiate additional
filings in other countries in 2017.

Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ: IONS), a
leader in antisense therapeutics. Biogen and Ionis conducted an
innovative clinical development program that moved SPINRAZA from its
first dose in humans in 2011 to its first regulatory approval in five
years. Based on the EC authorization of SPINRAZA, Ionis will receive a
$50 million milestone payment. Ionis is also eligible to receive tiered
royalties on global sales of SPINRAZA up to a percentage in the mid
teens.

About SMA 5-9

Spinal muscular atrophy (SMA) is characterized by loss of motor neurons
in the spinal cord and lower brain stem, resulting in severe and
progressive muscular atrophy and weakness. Ultimately, individuals with
the most severe type of SMA can become paralyzed and have difficulty
performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect in, the SMN1 gene, people with
SMA do not produce enough SMN protein, which is critical for the
maintenance of motor neurons. The severity of SMA correlates with the
amount of SMN protein. People with Type 1 SMA, the form that requires
the most intensive and supportive care, produce very little SMN protein
and do not achieve the ability to sit without support or live beyond two
years without respiratory support. People with Type 2 and Type 3 SMA
produce greater amounts of SMN protein and have less severe, but still
life-altering forms of SMA.

About SPINRAZA® (nusinersen)

SPINRAZA is being developed globally for the treatment of SMA.

SPINRAZA is an antisense oligonucleotide (ASO), using Ionis
Pharmaceuticals’ proprietary antisense technology, that is designed to
treat SMA caused by mutations or deletions in the SMN1 gene
located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA
alters the splicing of SMN2 pre-mRNA in order to increase
production of full-length SMN protein.10 ASOs are short
synthetic strings of nucleotides designed to selectively bind to target
RNA and regulate gene expression. Through use of this technology,
SPINRAZA has the potential to increase the amount of full-length SMN
protein in individuals with SMA.

About Biogen

Through cutting-edge science and medicine, Biogen discovers, develops
and delivers innovative therapies worldwide for people living with
serious neurological and neurodegenerative diseases. Founded in 1978,
Biogen is a pioneer in biotechnology and today the Company has the
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first and only approved treatment for spinal muscular
atrophy, and is at the forefront of neurology research for conditions
including Alzheimer’s disease, Parkinson’s disease and amyotrophic
lateral sclerosis. Biogen also manufactures and commercializes
biosimilars of advanced biologics. For more information, please visit www.biogen.com.
Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

Biogen Safe Harbor

This press release contains forward-looking statements, including
statements made pursuant to the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995 relating to the potential
benefits, safety and efficacy of SPINRAZA, the results of certain
real-world data, the status of current regulatory filings, plans for
additional regulatory filings in other jurisdictions, planning and
timing for commercial launch, and availability of patient access and
reimbursement pathways, which may vary on a country-by-country basis.
These forward-looking statements may be accompanied by words such as
“anticipate,” “believe,” “could,” “estimate,” “except,” “forecast,”
“intend,” “may,” “plan,” “potential,” “possible,” “will” and other words
and terms of similar meaning. You should not place undue reliance on
these statements or the scientific data presented. Drug development and
commercialization involve a high degree of risk. These statements
involve risks and uncertainties that could cause actual results to
differ materially from those reflected in such statements, including
without limitation uncertainty of success in commercialization of
SPINRAZA, which may be impacted by, among other things, the level of
preparedness of healthcare providers to treat patients, difficulties in
obtaining or changes in the availability of reimbursement for SPINRAZA,
the effectiveness of sales and marketing efforts, problems with the
manufacturing process for SPINRAZA, the occurrence of adverse safety
events, unexpected concerns that may arise from additional data or
analysis; regulatory authorities may require additional information or
further studies, or may fail to approve or may delay approval of
Biogen’s drug candidates or expansion of product labeling; or Biogen may
encounter other unexpected hurdles which may be impacted by, among other
things, the occurrence of adverse safety events, failure to obtain
regulatory approvals in certain jurisdictions, failure to obtain
regulatory approvals in other jurisdictions, failure to protect
intellectual property and other proprietary rights; product liability
claims; or third party collaboration risks. The foregoing sets forth
many, but not all, of the factors that could cause actual results to
differ from our expectations in any forward-looking statement. Investors
should consider this cautionary statement, as well as the risk factors
identified in Biogen’s most recent annual or quarterly report and in
other reports Biogen has filed with the U.S. Securities and Exchange
Commission. These statements are based on our current beliefes and
expectations and speak only as of the date of this press release. We do
not undertake any obligation to publicly update any forward-looking
statements, whether as a result of new information, future developments
or otherwise.